latest news

RBC Capital believes that Phase II PEG-PAL data and Phase I Achondroplasia safety data could be announced next week, 

http://wallstcheatsheet.com/stocks/biomarin-retains-outperform-rating-and-4-stock-analyses-sparking-chatter.html/

Q&A session

I am excited to start the online Q&A sessions. We have Dr. Wilcox signed up for Oct 30 at 4pm. I will send out details. We have Dr. Ain schedule for early 2012. I am trying to get more doctors to sign-up.

Just completed brochure and PR for LPA Dwarfism Awareness month event. I am flying to NYC this weekend for the event. 

Now that summer is over we will start ramping up activities and motivate more parents to raise $

Getting ready for the Grand Fando on Sept 16

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great comment

I wanted to share a comment from facebook in response to BioMarin's drug. I hope future generations do not have to endure what most little people have to experience today in terms of medical treatments

Amer

This is really interesting and complicated for me to process. I'm a mom to a young child with Achondroplasia as well as a woman with Achondroplasia who just went through two very complicated surgeries on my cervical spine. While goi

ng through the process I couldn't help but hope that my son will NEVER have to go through some of the procedures I did. But the unknown...is even scarier. Such a hard decision but could be a really good thing in the end. I wish there was a way to test it out without using our children in the trial runs...but I guess that's impossible? Thanks for sharing!

Time is flying. It has been over a month since I update the blog. I will write more about the LPA national convention. We had a great time and learned a lot. For now let me share an update from BioMarin. Nothing spectacular but good progress in the right direction.

http://seekingalpha.com/article/772391-biomarin-pharmaceutical-s-ceo-discusses-q2-2012-results-earnings-call-transcript?page=6

http://seekingalpha.com/article/772391-biomarin-pharmaceutical-s-ceo-discusses-q2-2012-results-earnings-call-transcript?page=7

Regards
Amer

Got approvals from doctors to use quotes. Exciting stuff.


uploaded new ambassador program
http://www.growingstronger.org/become-an-ambassador.html

getting ready for LPA nationals.  I hope we signup a lot of ambassadors

Below is an update I recently shared with a donor.

1. We found Sugen SU10270 within Pfizer and got the structure. Dr Wilcox and Dr Horton are testing the compound

2. We have published the description of the target molecule we are looking for.  Please share the link below with your contacts. 

https://docs.google.com/document/d/1b2zzFbo7bhC_bPkV8Uj0YFfMITgq3S-5H6wunYS0dxg/edit

3. We had a gala with presentations from Dr. Horton, Dr. Wilcox and friends who have dwarfism

See videos at: http://www.growingstronger.org/videos-and-presentations.html

4. We are a sponsor at the LPA National this year in Dallas. We will have a booth and meet and greet right after the "Research Panel" led by the LPA MAB (medical advisory board)

5. We are building a Parent advocate board so parents can sign-up to help fund raise and support each other

6. We are going to try and start 2 new online medical support programs

 - Online radio talk show with a MAB expert

 - Recruit MAB members to sign-up on http://www.healthtap.com 

7. We have raised $180K/year. We are just $20K/year short of our goal of $200K/year for 3 years to support Dr. Horton and Dr. Wilcox